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Synonyms: ION-363 | ION363 | Jacifusen
Compound class:
Nucleic acid
Comment: Ulefnersen (ION363) is an antisense oligonucleotide (ASO) that is designed to reduce the expression of FUS RNA binding protein (FUS). The P525L FUS variant is associated with the development of a rare and rapidly progressing juvenile form of amyotrophic lateral sclerosis (FUS-ALS) [1]. Antisense-mediated silencing of FUSP525L expression is proposed as a novel mechanism to reduce and/or reverse motor neuron loss and disease progression in FUS-ALS patients [2]. Ulefnersen is non-allele-specific, so inhibits expression of mutated and wild type FUS proteins.
Nucleotide sequence is: (3'-5')Gmoe=m5Cmoe-Amoe-Amoe-m5Umoe-dG=dT=m5Cd=dA=m5Cd=m5Cd=dT=dT=dT=m5Cd= Amoe-m5Umoe-Amoe=m5Cmoe=m5Cmoe, where - represents -PO(OH)-, = represents -PS(OH)-, Nmoe is 2'-O-(2-methoxyethyl)-nucleoside, m5N is 5-methyl-nucleoside and dN is 2'-deoxy-nucleoside. We don't have the full chemical structure for ulefnersen. |
Classification  |
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| Compound class | Nucleic acid |
| Compound subclass | Antisense oligonucleotide (ASO) |
| Target | FUS RNA binding protein (FUS) |
| International Nonproprietary Names |
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| INN number | INN |
| 12354 | ulefnersen |
| Synonyms |
| ION-363 | ION363 | Jacifusen |
| Database Links |
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| CAS Registry No. | 2589926-25-8 (source: WHO INN record) |
| ChEMBL Ligand | CHEMBL5095349 |
| DrugBank Ligand | DB18677 |
| GtoPdb PubChem SID | 491299838 |
| Search PubMed clinical trials | ulefnersen |
| Search PubMed titles | ulefnersen |
| Search PubMed titles/abstracts | ulefnersen |
